The biotechs Vertex and CRISPR Therapeutics have an interesting relationship as biotechs. They are partners are multiple levels but also are very different as companies including in size.
There’s been a key development in one of their partnerships.
Before we jump into that, please check out the video version of my 20 stem cell and regenerative medicine predictions for 2024 below. Subscribe to our YouTube channel too as we head for 1,000+ subscribers!
What’s up with Vertex and CRISPR Therapeutics?
“The big biotech has chosen to opt out of the diabetes gene-edited stem cell therapy it gained through the acquisition of ViaCyte, leaving CRISPR to take the clinical-phase program forward itself.”
“Vertex also remains active in diabetes cell therapy, including in partnership with CRISPR. In March, Vertex paid $100 million for non-exclusive rights to its partner’s CRISPR-Cas9 technology in hypoimmune cell therapies for type 1 diabetes. CRISPR received a further $70 million from Vertex later in the year and is still in line to receive up to $160 million in R&D milestones.”
Arnold Caplan death
The stem cell field lost one of the early pioneers recently when Professor Arnold Caplan passed away on Jan. 10. Caplan was considered the father of the mesenchymal stem cell or MSC field. He and others later felt that the MSC acronym should not be defined simply as stem cells since the cultures are somewhat heterogeneous. Other meanings for the MSC acronym began to be used.
I got to know him a little bit over the years and some good conversations. I had a long interview with him for The Niche about a decade ago. We even had some disagreements related to stem cell clinics marketing MSCs. He was a great person to talk with about anything related to stem cells. Arnold was the kind of person you felt would just be around forever and him dying at 82 is a bit of a shock. It seems way too young.
More recommended reads
- Scalise temporarily leaves Washington for stem cell treatment, WaPo. This is a bone marrow transplant-type procedure for a blood cancer.
- One of the researchers on the xenobots saw my newer post on anthrobots and gave me a heads-up on an article that the various xenobot authors all contributed to. They express some diverse views on how this all played out, which is really interesting: Biological Robots: Perspectives on an Emerging Interdisciplinary Field, Soft Robotics.
- Regulatory T cells in skin mediate immune privilege of the hair follicle stem cell niche, Science Immunology.
- Hallmarks of stemness in mammalian tissues, Science Immunology.
- A nonneural miRNA cluster mediates hearing via repression of two neural targets, G&D. A reader of The Niche pointed out that we haven’t covered cell therapy research for hearing impairment, which is a good point. I’m going to be writing about that sometime soon.
- Ancient DNA helps trace multiple sclerosis origins in European descendants, WaPo. My favorite line from this article from Carolyn Y. Johnson is “But genes don’t do just one thing.” I like it because so many folks oversimplify genes and, in particular, how they will gene edit them to supposedly yield only one desired result. Genetics and biology don’t work that simple way. In the case of MS predisposition genes, the same genes may protect against some pathogens. It’s akin to how variants that are somewhat protective against malaria can cause sickle cell. Some variants of the CCR5 gene that protect against HIV infection may make things worse when a person faces West Nile virus or influenza.
- Molecular basis for PHF7-mediated ubiquitination of histone H3, G&D.
Arnold Caplan (born Jan. 5, 1942 to Jan. 10, 2024) who discovered the MSC had an important affect on my life. Since 2018 I have been studying the secretome, which for my father had saved his life. My father had blood sepsis for nearly 2 years. All the standard medication did nothing to help my father. After one single treatment, my father had a blood test 9 days later at Watford General, his blood sepsis had gone. That was 2019 and the sepsis has not returned. RIP Arnold Caplan
Bill Jones, thanks for correcting my error (“medicinal stem cells”). Yes, “medicinal signaling cells,” and more to the point of the effect of this promotion in terminology to obscure the biologically significant cell heterogeneity of the preparations.
Kind regards,
James
My deepest condolences, he was a dear friend and colleague!
Tom ichim
Dear Admin:
We are advised to speak well of the dead, especially the recently dead; and I am sure that many praising eulogies for Caplan by his trainees and colleagues will follow soon in elitist research journals. But we should not speak well of the misdeeds, or if we are kinder, the missteps of the dead. This consideration is particularly applicable in the case of scientists like Caplan who are responsible for impeding progress in scientific knowledge and its medical development.
Now that Caplan is no longer with us, perhaps the field of mesenchymal stem cell research can achieve a more physiologically valid representation of important mesenchymal tissue cell preparations. My interactions with Caplan were through reading his scientific writings, attending his more recent virtual research conferences which he sponsored, and occasional exchanges between him and me, as a member of audiences, when he pontificated in his conference presentations. He was oppressive of other ideas from his position of prominence in the field, which to his is credit, he was a major founder and contributor. In my experiences with him, he gave little consideration to ideas that differed from his own and actively suppressed other perspectives.
Caplan publicly pronounced that his claim of having named mesenchymal tissue cell preparations to be “mesenchymal stem cells” was a mistaken appellation in light of subsequent studies showing the evident cell heterogeneity of these cell preparations. However, in the meantime, he destined this field of investigation, including professional science organizations like the International Society for Cell Therapy (ISCT), to treat these cell-heterogeneous preparations as if they were homogeneous for stem cells.
Once admitting his error, Caplan proceeded to drive the field into turmoil with a next extreme opinion of his own. He promoted either calling the cells “medicinal stem cells,” which would ignore the important cellular biology of the cell preparations altogether; or “mesenchymal stromal cells,” with which he made a worse error for stem cell science and medicine by throwing the critical stem cells in the preparations out with his self-cleansing bath water.
I hope that in the coming lionizations of the death of Caplan that the actual cellular biology of mesenchymal tissue cells is not further obscured by the same type of politics in science that investigators like Caplan deployed to promote their own ideas above the broader and richer work and ideas of the greater scientific research community.
James@Asymmetrex
Caplan called them “Medicinal Signaling Cells”
“I now urge that we change the name of MSCs to Medicinal Signaling Cells to more accurately reflect the fact that these cells home in on sites of injury or disease and secrete bioactive factors that are immunomodulatory and trophic (regenerative) meaning that these cells make therapeutic drugs in situ that are medicinal.”
https://stemcellsjournals.onlinelibrary.wiley.com/doi/10.1002/sctm.17-0051